Clinical trials: Spino Cerebellar Degeneration
Symptomatic Neurogenic Orthostatic Hypotension (NOH), Parkinson's Disease
Fipamezole in Neurogenic Orthostatic Hypotension
The purpose of this study is to determine whether Fipamezole is effective in the treatment of orthostatic hypotension and related symptoms in multiple system atrophy and Parkinson's disease.
- Conditions
- Symptomatic Neurogenic Orthostatic Hypotension (NOH), Parkinson's Disease, Multiple System Atrophy
- Trial name
- Foehn (NCT00758849)
- Testing
- Fipamezole
- Where
- 4 sites in France, Portugal
- Volunteers
- 24 people
- Sponsor
- Juvantia Pharma Ltd
Spinocerebellar Ataxia 3
Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3
There are no clinically established treatments which have been proven to delay the disease progression in spinocerebellar ataxia (SCA) 3.
- Trial name
- NCT04426149
- Testing
- trehalose
- Where
- Malaysia
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- National University of Malaysia
Friedreich Ataxia
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
- Trial name
- MOVE-FA (NCT04577352)
- Testing
- Vatiquinone
- Where
- 14 sites in United States, Australia and 7 more
- Volunteers
- 146 people
- Sponsor
- PTC Therapeutics
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Hereditary Ataxia, Spinocerebellar Degenerations
Core Stability Exercises and Hereditary Ataxia
The hereditary ataxias are a group of genetic disorders characterized by slowly progressive incoordination of gait and balance impairments in sitting and standing.
- Conditions
- Hereditary Ataxia, Spinocerebellar Degenerations, Cerebellar Ataxia
- Trial name
- Core-ataxia (NCT04750850)
- Testing
- Therapeutic core stability exercises, Usual care
- Where
- Spain
- Volunteers
- 20 people
- Sponsor
- Universitat Internacional de Catalunya
Friedreich's Ataxia
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.
- Trial name
- PROVE-FA (NCT07681713)
- Testing
- Vatiquinone
- Where
- 9 sites in United States, Belgium and 4 more
- Volunteers
- 120 people
- Sponsor
- PTC Therapeutics
Spinocerebellar Ataxia Type 3, Machado Joseph Disease
Safety and Efficacy of Lithium Carbonate in Patients With Spinocerebellar Ataxia Type 3
Design: Phase II-III, double-blind, parallel, placebo controlled randomized Clinical trial Background: Spinocerebellar ataxia type 3 (SCA-3) is an autosomal dominant adult-onset neurodegenerative disorder for which there is no…
- Trial name
- NCT01096082
- Testing
- Lithium Carbonate
- Where
- Brazil
- Volunteers
- 62 people
- Sponsor
- Hospital de Clinicas de Porto Alegre
Autosomal Dominant Spinocerebellar Ataxia (SCA1, 2,3,6,7,27B)
Autosomal Dominant Spinocerebellar Ataxias and Social Cognition
Spinocerebellar ataxias are a group of rare neurodegenerative diseases, clinically and genetically highly heterogeneous, with an estimated mean prevalence of 2.7 per 100,000 population.
- Trial name
- SoCoSca (NCT07099651)
- Testing
- tests
- Where
- France
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- University Hospital, Angers
Friedreich's Ataxia
A First in Human Study of RT001 in Patients With Friedreich's Ataxia
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia.
- Trial name
- NCT02445794
- Testing
- Low dose cohort, High dose cohort
- Where
- 2 sites in United States
- Volunteers
- 19 people
- Sponsor
- Biojiva LLC
Friedreich Ataxia
Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
To evaluate the safety and tolerability of single ascending doses of CTI-1601 in participants with Friedreich's ataxia
- Trial name
- NCT04176991
- Testing
- CTI-1601
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Larimar Therapeutics, Inc.
Spinocerebellar Ataxia
Wearable Gait Sensors
This research study is testing body-worn sensors to measure movement during simple tests of coordination, in order to evaluate the progression and severity of Spinocerebellar ataxia.
- Trial name
- NCT06628934
- Where
- United States
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- University of Chicago
Spinocerebellar Degeneration
An Extension Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)
The purpose of this study is to evaluate the long-term safety, efficacy, and pharmacokinetics of KPS-0373 in SCD patients.
- Trial name
- NCT01970111
- Testing
- KPS-0373, High dose, KPS-0373, Low dose
- Where
- Japan
- Sponsor
- Kissei Pharmaceutical Co., Ltd.
Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia 3
Clinical Trial Readiness for SCA1 and SCA3
The investigators plan to fill the gap between the current state of clinical trial readiness and the optimal one for SCA1 and SCA3, which are fatal rare diseases with no treatments.
- Trial name
- READISCA (NCT03487367)
- Where
- United States
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- The Methodist Hospital Research Institute
Friedreich Ataxia
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
The primary objective is to evaluate the potential effectiveness of an individualized intensive rehabilitation intervention using the "Prokin 252" stabilometric platform in the treatment of adolescent and adult patients with…
- Trial name
- NCT06692296
- Testing
- Balance training over stabilometric platform, Balance training by means of conventional rehabilitation
- Where
- Italy
- Volunteers
- 24 people
- Sponsor
- IRCCS Eugenio Medea
Hereditary Ataxia
Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia
The Hereditary Ataxias are a group of genetic disorders characterized by slowly progressive incoordination of gait and often associated with poor coordination of hands, speech, and eye movements.
- Trial name
- NCT01360164
- Testing
- human umbilical cord mesenchymal stem cells
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Shenzhen Beike Bio-Technology Co., Ltd.
Friedreich Ataxia, Cardiomyopathies
Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
The purpose of this study is to test the safety and preliminary efficacy of AAVrh.10hFXN to treat the cardiomyopathy associated with Friedreich's ataxia (FA).
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Cardiac Hypertrophy, Myocardial Fibrosis
- Trial name
- NCT05302271
- Testing
- AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN, Prednisone
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Weill Medical College of Cornell University
Friedreich Ataxia
Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA)
This dose-escalation study is aimed at investigating a novel application for artesunate in the treatment of Friedreich ataxia. It will evaluate this novel application of oral artesunate using a surrogate biological marker as…
- Trial name
- ARTEMIS (NCT04921930)
- Testing
- Artesunate Oral Product
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Friedreich Ataxia, Cardiomyopathy
Clinical Course Of Disease In Participants With FA-CM
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
- Trial name
- NCT06865482
- Where
- 19 sites in United States, Brazil and 6 more
- Volunteers
- 65 people
- Sponsor
- Lexeo Therapeutics
High Tumor Mutation Burden, High TMB (Tumor Mutation Burden)
Study of MK-3475 (Pembrolizumab) in Patients With Microsatellite Unstable (MSI) Tumors (Cohort D)
This study will be looking at whether MK-3475 (pembrolizumab) is effective (anti-tumor activity) and safe in patients with MSI (Microsatellite Unstable) negative cancer with a mutator phenotype.
- Conditions
- High Tumor Mutation Burden, High TMB (Tumor Mutation Burden), MSS (Microsatellite Stable)
- Trial name
- NCT04098068
- Testing
- MK-3475
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Metastatic Colorectal Cancer
Safety and Efficacy of Pembrolizumab (MK-3475) Plus Binimetinib Alone or Pembrolizumab Plus Chemotherapy With or Without Binimetinib in Metastatic Colorectal Cancer (mCRC) Participants (MK-3475-651/KEYNOTE-651)
The purpose of this study is to determine safety and tolerability and to establish a preliminary recommended Phase 2 dose (RP2D) for the following combinations: pembrolizumab plus binimetinib (Cohort A), pembrolizumab plus…
- Trial name
- NCT03374254
- Testing
- Pembrolizumab, Binimetinib, Oxaliplatin
- Where
- 14 sites in United States, Canada
- Volunteers
- 116 people
- Sponsor
- Merck Sharp & Dohme LLC